Evidence map›Paper›PMID 41614378›Full record

Trial reportEuropean journal of haematology2026

Efficacy and Safety of Prophylaxis With a Plasma-Derived von Willebrand Factor/Factor VIII Concentrate (Wilate) in Patients With Type 3 von Willebrand Disease-A WIL-31 Study Sub-Analysis.

Claudia Djambas Khayat, Leonid Dubey, Adlette Inati, Toshko Lissitchkov, Dzmitry Novik, Elina Peteva, Robert F Sidonio, Ali T Taher, Kateryna V Vilchevska, Vladimir Vdovin and 1 more

Registry-linked trialAbstract readClinical Trial, Phase IIIMulticenter Study
PubMed Full text at PMC Publisher
In one paragraph

Trial report in European journal of haematology, 2026. The graph could read no effect estimate from its abstract, so it casts no vote on the map. It is linked to trial NCT04052698 (Clinical Study to Investigate the Efficacy and Safety of Wilate During Prophylaxis in Previously Treated Patients With Von Willebrand Disease), which is not on this map. Not yet cited in PubMed.

0numbers the graph read from it
0cells of the map it votes in
0citing papers in PubMed
–field-weighted citation impact
1 · What the graph read from it

What it found

Each row is one number read from the abstract, on the scale the paper reported it, with its interval. Left of the dashed line favours the treatment, right favours the comparator. Under each row is the sentence it came from. New to these charts? A ten-minute tutorial.

The abstract states no effect estimate the extractor could read, or names no intervention and outcome on the map, so this paper lights no cell and moves no belief. It is still indexed, cited and linked below.

2 · The registry

The trial behind it

Trials whose registry record cites this paper, or whose number appears in the abstract. A trial that started after this paper was published is citing it as background, not reporting it.

NCT04052698 phase3completednot on this map

Clinical Study to Investigate the Efficacy and Safety of Wilate During Prophylaxis in Previously Treated Patients With Von Willebrand Disease (VWD)

TypeinterventionalSponsorOctapharmaRan2020 to 2022Enrolled43ConditionsVon Willebrand DiseasesArmsWilate
3 · Its place in the literature

Who cites it

0 citing papers in PubMed.

No citing paper in PubMed yet.

4 · The record

Corrections and comments

PubMed lists nothing against this paper. Absence here is not a guarantee, only a check that was made.

5 · Who and what money

Authors and funding

11 authors.

Claudia Djambas KhayatHotel Dieu de France Hospital, Saint Joseph University, Beirut, Lebanon.
Leonid DubeyCommunal Nonprofit Enterprise "Western Ukrainian Specialized Children's Medical Center" of Lviv Regional Council, Lviv, Ukraine.
Adlette InatiNINI Hospital and LAU Gilbert and Rose-Marie Chaghoury School of Medicine, Byblos and Beirut, Lebanon.
Toshko LissitchkovDepartment of Chemotherapy, Hemotherapy and Hereditary Blood Diseases at Clinical Hematology Clinic, Specialized Hospital for Active Treatment of Hematological Diseases, Sofia, Bulgaria.
Dzmitry NovikGovernment Agency "Republican Research Center for Radiation Medicine and Human Ecology", Gomel, Belarus.
Elina PetevaPediatric Clinic of Haematology and Oncology, University Hospital St. Marina-Varna, Varna, Bulgaria.
Robert F SidonioDepartment of Pediatrics, Emory University School of Medicine, Atlanta, Georgia, USA.ORCID https://orcid.org/0000-0002-9509-9415
Ali T TaherDepartment of Internal Medicine, Division of Hematology-Oncology, American University of Beirut Medical Center, Beirut, Lebanon.
Kateryna V VilchevskaNational Specialized Children's Hospital Okhmatdyt, Center of Hemostasis Pathologies, Kyiv, Ukraine.
Vladimir VdovinMorozovskaya Children's City Clinical Hospital, Moscow, Russia.
Ana BobanDepartment of Internal Medicine, University Hospital Centre Zagreb, Zagreb, Croatia.

Funding

Octapharma AG
6 · The paper itself

Abstract

objectivesThe WIL-31 study demonstrated efficacy and safety of prophylaxis with the plasma-derived von Willebrand factor/factor VIII concentrate wilate in von Willebrand disease (VWD) of all types and was the only prospective study with an on-demand run-in study as an intra-individual comparator. This subgroup analysis examines the efficacy of wilate prophylaxis in patients with type 3 VWD in WIL-31.

methodsPatients received 20-40 IU/kg wilate prophylaxis 2-3 times weekly for 12 months.

resultsTwenty-two patients in WIL-31 had type 3 VWD. Mean total annualized bleeding rate (ABRs) during on-demand versus prophylaxis was 37.1 versus 5.2 (86% reduction). During prophylaxis, 115 bleeds occurred, most (95%) of which were minor; the most common bleeding site was the nose (40% of bleeds). Mean overall spontaneous ABRs during on-demand versus prophylaxis were 26.5 versus 2.8 (89% reduction); mean treated spontaneous ABRs were 20.3 versus 1.4, respectively (93% reduction). ABRs were reduced further during the second 6 months of prophylaxis versus the first 6 months. Results were consistent in subgroups by age. No serious adverse events related to study treatment and no thrombotic events were observed.

conclusionsProphylaxis with wilate was effective and well tolerated in patients with type 3 VWD, in all age groups.

trial registrationNCT04052698; https://clinicaltrials.gov/study/NCT04052698.

Indexed as

Factor VIIIvon Willebrand Disease, Type 3von Willebrand FactorAdolescentAdultAgedChildChild, PreschoolDrug CombinationsFemaleHemorrhageHumansInfantMaleMiddle AgedTreatment OutcomeDrug CombinationsFactor VIIIfactor VIII, von Willebrand factor drug combinationvon Willebrand Factorhemostaticsprevention & controltype 3 von Willebrand diseasevon Willebrand diseasevon Willebrand factor

Identifiers

PMID41614378
PMCPMC13050673

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Registered trials

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